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A Phase 1/ Phase 2 Study of TTHX1114(NM141) (INTREPID)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04520321
Recruitment Status : Recruiting
First Posted : August 20, 2020
Last Update Posted : September 25, 2020
Information provided by (Responsible Party):
Trefoil Therapeutics, Inc.

Brief Summary:
Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study that includes a non-interventional observational sub-study in which subjects will undergo (standard) ocular assessments

Condition or disease Intervention/treatment Phase
Corneal Endothelial Dystrophy Fuchs Endothelial Corneal Dystrophy Pseudophakic Bullous Keratopathy Endothelial Dysfunction Drug: TTHX1114(NM141) Other: Vehicle (placebo) Phase 1 Phase 2

Detailed Description:
This is a prospective, multi-center, randomized, masked, vehicle-controlled, dose-escalation study that will include an observational (no intervention) sub-study. Up to 71 eligible subjects with moderate to severe corneal endothelial dystrophy (defined as Endothelial Cell Density < 2000 mm^2) in at least one eye will be enrolled and randomized in the main study. The Observational Sub-study will enroll approximately 25 to 50 subjects.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 71 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Randomized, Masked, Vehicle-controlled, Dose-escalation study
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1/ Phase 2 Study Evaluating the Safety and Efficacy of the Investigational New Drug TTHX1114(NM141) on the Regeneration of Corneal Endothelial Cells in Patients With Corneal Endothelial Dystrophies Following Intracameral Delivery
Actual Study Start Date : August 19, 2020
Estimated Primary Completion Date : August 2021
Estimated Study Completion Date : December 2021

Arm Intervention/treatment
Placebo Comparator: Vehicle (placebo)
Placebo weekly x 4
Other: Vehicle (placebo)

Experimental: Low dose
TTHX1114(NM141) low-dose weekly x 4
Drug: TTHX1114(NM141)
engineered FGF-1 delivered intracamerally

Experimental: Mid-dose
TTHX1114(NM141) mid-dose weekly x 4
Drug: TTHX1114(NM141)
engineered FGF-1 delivered intracamerally

Experimental: High-dose
TTHX1114(NM141) high-dose weekly x 4
Drug: TTHX1114(NM141)
engineered FGF-1 delivered intracamerally

Primary Outcome Measures :
  1. Change in Corneal Endothelial Cell Count [ Time Frame: 56 Days ]
    Measured with Specular Microscopy

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • Fuchs Endothelial Corneal Dystrophy, pseudophakic bullous keratopathy, or endothelial dysfunction/ insufficiency due to surgical intervention diagnosed more than 6 months prior to Study Day 0
  • Central endothelial cell count of < 2000 mm^2 in at least one eye as determined by the central reading facility

Key Exclusion Criteria:

  • Conditions that would impair examination of the anterior chamber structure
  • Documented repeated elevated intra ocular pressure (in either eye)
  • Corneal transplant (in either eye)
  • Posterior Polymorphous Corneal Dystrophy (PPCD)
  • History of uveitis or herpetic keratitis
  • Cataract surgery within the past 3 months
  • Refractive surgery (in the Study Eye)
  • Anterior Chamber IOL placement (in the Study Eye)
  • Active extra-ocular inflammation from any non-infectious or infectious cause within the past 6 months
  • Expected or planned ocular surgery within the next 3 months
  • Use of cytotoxic chemotherapy within the last 1 month
  • Treatment with a rho kinase inhibitor within the last 3 months
  • Use of cyclosporine ophthalmic emulsion or lifitegrast ophthalmic solution in the last 30 days
  • Systemic or ophthalmic corticosteroid use in the 30 days prior to Study Day 0 unless approved by the Medical Monitor
  • History of significant allergy, hypersensitivity, or intolerance to any drug compound, food, or other substance
  • Unwilling to use birth control

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04520321

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Contact: Thomas M Tremblay, RN BSN (415) 305-3491

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United States, California
North Bay Eye Associates, Inc. Recruiting
Petaluma, California, United States, 94954
Contact: Eduardo Chavez    707-769-2240      
United States, Florida
Levenson Eye Associates, Inc Recruiting
Jacksonville, Florida, United States, 32204
Contact: Vontrece Williams    904-701-2417      
United States, Illinois
Chicago Corneal Consultants Recruiting
Hoffman Estates, Illinois, United States, 60169
Contact: Parag Majmudar, MD         
Contact    847-748-3553   
United States, Indiana
Price Vision Group Recruiting
Indianapolis, Indiana, United States, 46260
Contact: Marianne Price, PhD    317-814-2990   
United States, Missouri
Tauber Eye Center Recruiting
Kansas City, Missouri, United States, 64111
Contact: Megan Hefter         
Contact    816-531-9100   
United States, New York
Alterman, Modi and Wolter Recruiting
Poughkeepsie, New York, United States, 12603
Contact: Sarah Ortiz Piza         
Contact    845- 454-1025      
Sponsors and Collaborators
Trefoil Therapeutics, Inc.
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Study Director: Thomas M Tremblay, RN BSN Trefoil Therapeutics
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Responsible Party: Trefoil Therapeutics, Inc. Identifier: NCT04520321    
Other Study ID Numbers: TTHX-001
First Posted: August 20, 2020    Key Record Dates
Last Update Posted: September 25, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Iridocorneal Endothelial Syndrome
Fuchs' Endothelial Dystrophy
Corneal Dystrophies, Hereditary
Corneal Diseases
Eye Diseases
Iris Diseases
Uveal Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn